An Astellas Company

targeting mitochondriaadvancing human health

January 23, 2018

Astellas Completes Acquisition of Mitobridge, Inc.

Astellas Completes Acquisition of Mitobridge, Inc.Tokyo, Janurary 24, 2018 - Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, “Astellas” ) today announced that it has completed the acquisition of Mitobridge, Inc. (“Mitobridge”), and Mitobridge has become a wholly owned subsidiary of Astellas as of U.S. Eastern Time January 23, 2018.

By exercising the option right to acquire Mitobridge, Astellas paid $225 million1 to acquire 100% of the equity in Mitobridge. In addition, Mitobridge shareholders will be eligible for additional payments from Astellas that total up to $225 million2 depending on the progress of various programs in clinical development.

The transaction accelerates Astellas’ research and development in diseases associated with mitochondrial dysfunctions and will enable the delivery of innovative new treatment options to patients. At this point, Astellas will maintain the organization in Cambridge, MA, and retain the Mitobridge name as a division of Astellas.

Astellas is still reviewing the impact of the completion of the acquisition on its financial results for the fiscal year ending March 31, 2018.

As Astellas is a shareholder in Mitobridge, the actual payment by Astellas to Mitobridge shareholders after adjustment based on the estimate of cash and cash equivalents etc. held by Mitobridge is $161.7 The amount is subject to further true-up based on actual numbers as of the closing date.

As Astellas is a shareholder in Mitobridge, the actual payment by Astellas to Mitobridge shareholders will be $165.5 million.

About AstellasAstellas Pharma Inc., based in Tokyo, Japan, is a company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. We focus on Urology, Oncology, Immunology, Nephrology and Neuroscience as prioritized therapeutic areas while advancing new therapeutic areas and discovery research leveraging new technologies/modalities. We are also creating new value by combining internal capabilities and external expertise in the medical/healthcare business. Astellas is on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en.

Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

November 30, 2017

Astellas Acquires Mitobridge Under Existing Collaboration

Astellas Acquires Mitobridge Under Existing Collaboration

-Accelerating the Discovery and Development of Novel Drugs that Target Mitochondrial Functions -

Tokyo and Cambridge, November 30, 2017 - Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, “Astellas”) and Mitobridge, Inc.1 (President and CEO: Kazumi Shiosaki, “Mitobridge”) today announced that Astellas has exercised its exclusive option right to acquire Mitobridge under the amended terms of the October 2013 partnership agreement governing their an R&D collaboration focused on discovering and developing novel drugs that target mitochondrial function. Mitobridge will become a wholly-owned subsidiary of Astellas following the close of the acquisition.

Under the 2013 partnership agreements, Astellas and Mitobridge have collaborated to discover and develop compounds that target mitochondrial function. These drug candidates have the potential to treat genetic, metabolic or neurodegenerative disorders as well as conditions of aging. MA-0211, the most advanced program emerging from the collaboration, is currently in Phase 1 clinical studies for Duchenne Muscular Dystrophy (DMD).

“Astellas has increased the involvement in mitochondrial drug discovery through its partnership with Mitobridge and the network of key scientists that the company has assembled. The achievements resulting from the collaboration have exceeded our expectations including the rapid advancement of multiple programs,“ commented Yoshihiko Hatanaka, President and CEO, Astellas. “Through the acquisition of Mitobridge, Astellas accelerates our investment in diseases caused by mitochondria dysfunctions in order to deliver innovative new treatment options to patients.”

“We at Mitobridge are delighted to become part of Astellas’ major initiative to develop new therapies for serious diseases. We are grateful for the tremendous support and guidance from Astellas, MPM Capital, and Longwood Fund that enabled Mitobridge to rapidly build an exciting pipeline,” said Kazumi Shiosaki, Ph.D., President & CEO, and co-founder of Mitobridge. “Mitobridge – as a subsidary of Astellas – will now have additional resources to continue to advance and expand on its achievements to date.”

By exercising the option right according to the amended agreement, Astellas will pay an upfront fee of $225 million2 to acquire Mitobridge. Mitobridge will be eligible for additional payments that total up to $225 million2, depending on the progress of various programs in clinical development. The closing of the acquisition is subject to customary regulatory approvals, and is expected to be finalized in the next several weeks.

The impact of this transaction on Astellas’ financial results for the fiscal year ending March 31, 2018 will be immaterial.

(1) The name of Mitokyne, Inc. was changed to Mitobridge, Inc.

(2) As Astellas is a shareholder in Mitobridge, the actual payment by Astellas to Mitobridge will be $165.5 million. The amount assumes the current Astellas’ shares on a fully diluted basis. The amount will be adjusted depending on the cash and cash equivalents etc. held by Mitobridge at the closing.

(4) Amount:$225 million* to make Mitobridge a wholly-owned subsidiary of AstellasUp to $225 million* in future contingent payments based on the advances in clinical programs*As Astellas is a shareholder in Mitobridge, the actual payment by Astellas to Mitobridge will be $165.5 million. The amount assumes the current Astellas’ shares on a fully diluted basis. The amount will be adjusted depending on the cash and cash equivalents etc. held by Mitobridge at the closing.(5) Expected timing of closing: Next several weeks, subject to customary regulatory approvals

Astellas Pharma Inc., based in Tokyo, Japan, is a company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. We focus on Urology, Oncology, Immunology, Nephrology and Neuroscience as prioritized therapeutic areas while advancing new therapeutic areas and discovery research leveraging new technologies/modalities. We are also creating new value by combining internal capabilities and external expertise in the medical/healthcare business. Astellas is on the forefront of healthcare change to turn innovative science into value for patients. For more information, please visit our website at https://www.astellas.com/en.

About Mitobridge (fka Mitokyne)

Mitobridge is discovering and developing small molecule therapeutics that enhance mitochondrial function and that build upon the emerging scientific findings linking mitochondrial dysfunction with disease pathologies. The Company is progressing innovative approaches to the treatment of diseases with high unmet medical need with the strategy to establish proof of concept in rare diseases and then expand into more common diseases. The Company was founded in 2011 with seed financing from MPM Capital. In October 2013, it closed a Series A financing from MPM Capital, Longwood Fund, and Astellas Pharma and a shared vision for the promise of mitochondrial-targeted therapeutics.

Astellas Cautionary Notes

In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice.

The data, presented last week at the Annual Meeting of the American Society of Nephrology (ASN), demonstrated that Mitobridge’s proprietary compound MA-0217 (also known as MTB-2) corrects the mitochondrial deficits and alleviates the renal dysfunction in a rat ischemia reperfusion AKI (IR-AKI) model. Additionally, MA-0204, a close analog of MA-0217, dosed post IR-AKI in a rat model of patients with hypertension, chronic kidney disease and diabetes and thereby at high risk to develop AKI, reduced kidney injury, accelerated recovery of kidney function and decreased the onset of fibrosis and slowed progression of diabetes and chronic kidney disease by 4 weeks. The posters are available on the Company’s website:https://www.mitobridge.com/news/publications.

“AKI is a serious condition and there are no currently approved therapies to help manage patients,” said Bruce A. Molitoris, Professor of Medicine and Director of the Indiana Center for Biological Microscopy at Indiana University. “MA-0217 may address the cellular injury, mitochondrial dysfunction and organ damage associated with AKI and represents an innovative option for this life-threatening condition. The presented data are quite promising and I look forward to seeing the compound enter clinical development.”

About Mitobridge

Mitobridge is dedicated to delivering therapeutics that improve mitochondrial function. Our team of experienced drug discovery and development scientists is leveraging their exceptional knowledge of mitochondria biology to develop a pipeline of innovative programs for the treatment of kidney and muscle diseases with high unmet medical need. Headquartered in Cambridge, MA, Mitobridge was launched in October 2013 with funding from Astellas Pharma, Inc., MPM Capital and Longwood Ventures. For more information about the Company, please visit www.mitobridge.com.

About MA-0217

MA-0217 (MTB-2) is a potent and highly-selective PPARδ modulator and the second program to emerge from Mitobridge’s platform. The compound is a potentially first-in-class approach to treating acute kidney injury (AKI). Mitobridge has generated preclinical data demonstrating that intervention with MA-0217 improves mitochondrial function, overall energy metabolism and performance of the kidney following an acute ischemia reperfusion injury.

About AKI

AKI is a sudden loss of kidney function that often occurs in hospitalized patients as a result of cardiac and/or vascular surgery, trauma, infection, cardiac disease or being treated with nephrotoxic anti-cancer therapy. AKI affects more than 13 million people each year worldwide and is associated with extended hospitalization and increased mortality. Currently, there are no therapies to prevent or treat AKI. The clinical manifestations are, in part, due to early onset mitochondrial deficits that drive multiple pathophysiological events that lead to AKI and appear to be linked to the severity of AKI and progression to Chronic Kidney Disease (CKD).

September 18, 2017

Company Licenses Intellectual Property from École polytechnique fédérale de Lausanne

CAMBRIDGE, MA, September 18, 2017 -- Mitobridge, Inc., a biopharmaceutical company pioneering the discovery and development of products that improve mitochondrial function, broadens its therapeutic approach to include Nicotinamide Adenine Dinucleotide (NAD+) pathway modulation. The company entered into a license agreement with École polytechnique fédérale de Lausanne (EPFL) for intellectual property (IP) related to the treatment of a broad range of diseases as well as conditions of aging utilizing compounds that boost NAD+ levels. The company is advancing therapeutics for muscular dystrophies and myopathies as well as kidney, liver and neurodegenerative diseases that will benefit from improved mitochondrial function.

NAD+ is an essential coenzyme that plays a key role in metabolism, mitochondrial energy production and cell signaling. Numerous studies have demonstrated that reduced NAD+ levels are associated with mitochondrial dysfunction and that maintaining or raising NAD+ levels in cells that are under stress diminishes the deleterious effects. Compounds that protect or elevate NAD+ levels could provide therapeutic benefits in many medical and age-associated conditions. Mitobridge is developing drug candidates that are directed at multiple points in the NAD+ biosynthetic and metabolic pathway. Our approach to modulating NAD+ levels includes programs targeting Poly ADP Ribose Polymerase (PARP), Aminocarboxymuconate Semialdehyde Decarboxylase (ACMSD) and N′-Nicotinamide Methyltransferase (NNMT).

The exclusive worldwide license includes IP based on research from the laboratory of Johan Auwerx, MD, PhD. Dr. Auwerx, a leader in the field of NAD+ modulation, is a Professor at EPFL in Lausanne, Switzerland. “Modulating NAD+ levels represents an innovative strategy for improving mitochondrial function and holds great promise for therapeutic development. I am delighted that Mitobridge is pursuing this approach and look forward to working with the team to progress new product candidates,” stated Dr. Auwerx.

Mitobridge is employing several therapeutic strategies for improving mitochondrial function. The first compound to enter clinical development is MA-0211 (MTB-1), an orally bioavailable PPARd modulator being tested in healthy volunteers in preparation for a trial in Duchenne Muscular Dystrophy (DMD). As DMD is characterized by mitochondrial defects, inadequate energy supply and muscle fibrosis, intervening with MA-0211 may be therapeutically beneficial for all DMD patients, regardless of their underlying dystrophin mutation. Additional clinical studies of MA-0211 in other diseases characterized by mitochondrial defects are currently being planned.

About Mitobridge

Mitobridge is dedicated to delivering therapeutics that improve mitochondrial function. Our team of experienced drug discovery, translational and development scientists are leveraging their exceptional knowledge of mitochondria biology to deliver a pipeline of innovative programs for the treatment of muscle and kidney diseases as well as other serious medical conditions. Headquartered in Cambridge, MA, Mitobridge was launched in October 2013 with funding from Astellas Pharma Inc., MPM Capital and Longwood Ventures. For more information about the Company, please visit www.mitobridge.com.

CAMBRIDGE, MA, August 8, 2017 -- Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the initiation of the first-in-human Phase I trial of its PPAR-delta (PPARδ) modulator, MA-0211 (also known as MTB-1). The study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MA-0211 in healthy volunteers, which will provide the basis for a trial program in Duchenne Muscular Dystrophy (DMD) patients. MA-0211 is the first clinical compound to emerge from Mitobridge’s mitochondrial enhancement platform. The PPARδ modulator aims to reverse the mitochondrial deficits in DMD, which play a key role in disease progression.

DMD is a rare genetic disease caused by loss-of-function mutations in the dystrophin gene. This debilitating fatal disorder affects males and leads to progressive cardiac, skeletal and smooth muscle weakness and eventual loss of muscle mass. Mitobridge’s research has confirmed and expanded upon previous studies showing that mitochondrial defects contribute to abnormalities in the dystrophic muscle and play a central role in the etiology of DMD.

Mitobridge scientists have assembled extensive nonclinical data in patient samples and genetic animal models demonstrating that MA-0211 may be therapeutically beneficial to DMD patients. Treatment of DMD patient muscle cells with MA-0211 upregulated genes related to fatty acid oxidation, which increased mitochondrial function and mitochondrial biogenesis. MA-0211 was evaluated in the widely used DMD mouse model, the mdx mouse, which has a point mutation in the dystrophin gene and recapitulates many of the deficiencies seen in DMD patients. Once-daily oral dosing of MA-0211 for five weeks in mdx mice produced several therapeutic benefits including increased running endurance on a treadmill, decreased muscle necrosis and inflammation and decreased diaphragm fibrosis. In a similar study, six months of dosing in older mdx mice resulted in decreased serum creatine kinase and improved cardiac and respiratory function compared to untreated mdx mice. The strong pre-clinical data are the basis for advancing the compound into clinical development and highlight MA-0211’s potential to reverse key defects and slow disease progression. MA-0211 is being developed with the Company’s corporate partner, Astellas Pharma, Inc.

Recently, George Mulligan, Mitobridge’s SVP of Translation Medicine, presented an update of the MA-0211 program at the Parent Project Muscular Dystrophy Annual Connect Conference on June 30, 2017. The presentation is available here: https://youtu.be/leQnKrVm4YI

About MA-0211

MA-0211 (MTB-1), an orally bioavailable PPARδ modulator, is the first clinical compound to emerge from Mitobridge’s mitochondrial enhancement platform. In studies with patient muscle cells and animals, MA-0211 improves mitochondrial function, overall energy metabolism, muscle performance and regeneration. As DMD is characterized by mitochondrial defects, inadequate energy supply and muscle fibrosis, intervening with MA-0211 may be therapeutically beneficial for all DMD patients, regardless of their underlying dystrophin mutation. Additional clinical studies in other diseases characterized by mitochondrial deficiencies are currently being planned.

About Mitobridge

Mitobridge is dedicated to delivering therapeutics that improve mitochondrial function. Our team of experienced drug discovery and development scientists are leveraging their exceptional knowledge of mitochondria biology to develop a pipeline of innovative programs for the treatment of kidney and muscle diseases with high unmet medical need. Headquartered in Cambridge, MA, Mitobridge was launched in October 2013 with funding from Astellas Pharma, Inc., MPM Capital and Longwood Ventures. For more information about the Company, please visit www.mitobridge.com.

September 16, 2015

Mitobridge Announces Sponsored Research Agreement to Evaluate PPARδ Compounds in Cells from Patients with Mitochondrial Disease

Mitobridge Announces Sponsored Research Agreement to Evaluate PPARδ Compounds in Cells from Patients with Mitochondrial Disease

CAMBRIDGE, MA-- Mitobridge, Inc., a biopharmaceutical company pioneering the discovery and development of products that improve mitochondrial function, announces the signing of a sponsored research agreement to assess Mitobridge’s selective Peroxisome Proliferator-Activated Receptor Delta (PPARδ) modulators in mitochondrial disease patient cell lines. Mitobridge will support research in the laboratory of Dr. Marni J. Falk, Assistant Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, and Director of The Children’s Hospital of Philadelphia Mitochondrial-Genetic Disease Clinic. The aim of the collaboration is to evaluate the therapeutic effects of PPARδ modulators on mitochondrial function in fibroblasts derived from patients with genetically-defined mitochondrial disorders.

“We have a shared commitment to progressing innovative treatments for mitochondrial diseases, a group of disorders with tremendous unmet medical need,” commented Effie Tozzo, PhD, Vice President of Translational Biology at Mitobridge. “We are delighted to be collaborating with a leading mitochondrial disease clinician-scientist who has demonstrated expertise in translating her experience with patients into important scientific advancements.”

The research plan is designed to evaluate the ability of Mitobridge’s proprietary compounds to improve mitochondrial function in patient cells. The cells harbor specific disease-causing mutations in mitochondrial DNA and in nuclear genes that are essential for mitochondrial energy production. Positive results could support the clinical development of the PPARδ modulators for certain mitochondrial diseases such as MELAS and Leigh’s Syndrome. Earlier this year, Mitobridge convened a panel of key opinion leaders in the field including, Patrick Chinnery, MD, PhD, Richard Haas, MD, Gregory Enns, MD, Michio Hirano, MD, and Tanja Taivassalo, PhD, and they recommended that Mitobridge study the activity of PPARδ modulators on mitochondrial function in patient-derived cell lines.

Mitobridge believes that testing therapeutic candidates in Dr. Falk’s collection of fibroblast cell lines, which are derived directly from mitochondrial disease patients’ skin biopsies, should lead to a better understanding of the defects in these cells and the potential benefit for patients.

Members of Mitobridge Mitochondrial Myopathy Advisory Panel

Patrick Chinnery, MD, PhD, Professor of Neurology and Head of the Department of Clinical Neurosciences at the University of Cambridge, Richard Haas, MD, Professor of Neuroscience at UC San Diego (UCSD) School of Medicine and Director of the UCSD Mitochondrial Disease Laboratory, Gregory Enns, MD, Professor of Pediatrics (Medical Genetics) at Stanford University, Michio Hirano, MD, Professor of Neurology at Columbia University and Co-director of the North American Mitochondrial Disease Consortium (NAMDC) and Tanja Taivassalo, PhD, Associate Professor, Department of Kinesiology at McGill University.

About Mitobridge

Mitobridge is a leader in the emerging field of mitochondria-related drug discovery and development. The Company is utilizing its innovative screening platform to identify and develop novel therapeutics that improve mitochondria function and provide treatments for mitochondrial genetic diseases, musculoskeletal, kidney and neurodegenerative disorders as well as diseases and conditions of aging. Mitobridge’s scientific founders and advisors include world-renowned leaders in biology and mitochondrial function. For more information about Mitobridge, please visit the company's website at www.mitobridge.com

February 23, 2015

Mitobridge Secures Key Intellectual Property from the Salk Institute

–CAMBRIDGE, Mass., February 23, 2015 — Mitobridge, Inc., previously known as Mitokyne, Inc., a biopharmaceutical company pioneering the discovery and development of products that improve mitochondrial function, today announced that the Company has entered into an agreement with the Salk Institute for Biological Sciences to access intellectual property and technology related to Peroxisome Proliferator-Activated Receptor Delta (PPARδ). The exclusive worldwide license grants key patents and reagents developed in the laboratory of Mitobridge co-founder Professor Ronald Evans for the diagnosis, prevention and treatment of diseases in both humans and animals. Dr. Evans, who was bestowed the Lasker Award for his work in nuclear hormone receptors, is the March of Dimes Chair in Developmental and Molecular Biology and Professor and Director of the Gene Expression Laboratory at the Salk Institute. He is also a Howard Hughes Medical Institute investigator.

Mitobridge is advancing a selective PPAR modulator (SPPARM) program based on recent developments in the understanding of the pharmacology of this drug target and the potential to utilize a PPARδ modulator as an exercise mimetic. The Company’s latest results indicate that a PPARδ modulator may be working through mitochondria and have potential to treat mitochondrial myopathies as well as other musculoskeletal disorders.

“We are excited about the opportunity to capitalize on the innovative PPARδ technology that has been developed over the past several years in the Evans lab. The results from pre-clinical studies suggest that our molecules might be safer yet similarly effective to earlier generation PPARδ compounds”, commented Mike Patane, PhD, Senior Vice President of Drug Discovery at Mitobridge. “We hope to translate the original discoveries from the Evans lab into meaningful therapeutics for disorders that will benefit from improving mitochondrial function.”

The Company was launched in October 2013 under the name Mitokyne to capitalize on the emerging biological understanding of how mitochondria function impacts health via bioenergetics, signaling, dynamics and biosynthesis. The Company recently changed its name to Mitobridge and filed a trademark application for that name. In addition, Mitobridge has closed a second tranche of financing led by MPM Capital, Longwood Fund and Astellas Pharma Inc.

“I am pleased with the progress the Company has made in our first year and the commitment demonstrated by our investors and our therapeutics partner, Astellas Pharma,” said Kazumi Shiosaki, PhD, President and CEO of Mitobridge. “Considerable insight linking mitochondrial dysfunction and disease pathologies has been gained over the last few years, and Mitobridge is poised for a leadership position in discovering innovative therapies.”

About Mitobridge

Mitobridge is a leader in the emerging field of mitochondria-related drug discovery and development. The Company is utilizing its innovative screening platform to identify and develop novel therapeutics that improve mitochondria function and provide treatments for mitochondrial genetic diseases, musculoskeletal and neurodegenerative disorders as well as diseases and conditions of aging. Mitobridge’s scientific founders and advisors include world-renowned leaders in biology and mitochondrial function. For more information about Mitobridge, please visit the company's website at www.mitobridge.com

About the Salk Institute for Biological Studies

The Salk Institute for Biological Studies is one of the world's preeminent basic research institutions, where internationally renowned faculty probes fundamental life science questions in a unique, collaborative, and creative environment. Focused both on discovery and on mentoring future generations of researchers, Salk scientists make groundbreaking contributions to our understanding of cancer, aging, Alzheimer's, diabetes and infectious diseases by studying neuroscience, genetics, cell and plant biology, and related disciplines.

Faculty achievements have been recognized with numerous honors, including Nobel Prizes and memberships in the National Academy of Sciences. Founded in 1960 by polio vaccine pioneer Jonas Salk, MD, the Institute is an independent nonprofit organization and architectural landmark.

October 7, 2013

– Collaboration Will Focus on Expanding the New Frontier of Mitochondria-Related Disease –

Tokyo and Boston, October 7, 2013 --- Astellas Pharma Inc. (Tokyo Stock Exchange: 4503, President and CEO: Yoshihiko Hatanaka, hereinafter called “Astellas”) and Mitokyne, Inc. (President and CEO: Kazumi Shiosaki, hereinafter called “Mitokyne”) announced today that the two companies have entered into an exclusive R&D collaboration focused on discovering and developing novel drugs that improve mitochondrial functions. These drug candidates have the potential to treat genetic, metabolic or neurodegenerative disorders as well as conditions of aging. The emerging biological understanding of mitochondria, along with new tools and assays available to better elucidate mitochondrial function and processes, will enable the identification of breakthrough treatments for patients suffering from mitochondria-related diseases and improve their quality of life.

Concurrently, Mitokyne closed a $45M series A equity financing from Astellas, MPM Capital, and Longwood Founders Fund. In connection with the financing, Ansbert Gadicke, MD, MPM Capital, Rich Aldrich, Longwood Founders Fund, and a designee from Astellas joined the Mitokyne Board of Directors.

Under the collaboration agreement, Astellas and Mitokyne will collaborate to discover and develop compounds that target mitochondrial function. Mitokyne will lead all the research and drug discovery activities and be responsible for delivering IND candidates, whereupon Astellas will assume all clinical development activities as well as commercialization. In addition to generating a pipeline of novel drug candidates, Mitokyne is developing a novel mitochondrial screening platform to enhance ongoing R&D programs and identify new drug targets. Astellas has the exclusive right to acquire Mitokyne during certain periods during the term of the five– year agreement. Including upfront, R&D funding, acquisition and milestone payments, the total value of the partnership agreement could reach up to $730M. Taking into account Astellas’ participation in Mitokyne’s equity financing, Astellas’ actual payment for the acquisition based on equity in Mitokyne could be over $500M in accordance with conditions of the agreement.

“This R&D partnership is an excellent opportunity for Astellas to utilize more external capabilities and resources, and to undertake initiatives related to new therapeutic areas, as we announced “Reshaping Research Framework” in May,” said Yoshihiko Hatanaka, President & CEO of Astellas. “Through this partnership, we expect to pursue advanced drug discovery and establish a winning model with which it can solidify its position as the leader in the field of mitochondria-related diseases, and eventually develop advanced medical solutions for patients around the world, adding to our portfolio of innovative new drugs.”

Mitokyne’s scientific founders include world-renowned leaders in biology and mitochondrial function. Johan Auwerx is the Nestle Chair in Energy Metabolism and Professor at the cole Polytechnique Fdrale de Lausanne. Andrew Dillin is the Siebel Distinguished Chair of Stem Cell Biology, HHMI Investigator and Professor of Genetic, Genomics and Development at UC Berkeley. Ronald Evans is the March of Dimes Chair in Developmental and Molecular Biology and Professor and Director in the Gene Expression Laboratory at Salk Institute for Biological Studies and was awarded the Lasker Award for his work in nuclear hormone receptors. H. Robert Horvitz is Professor in the Department of Biology and a member of the McGovern Institute for Brain Research and of the Koch Institute for Integrative Cancer Research at the Massachusetts Institute of Technology. Professor Horvitz received the Nobel Prize in 2002 for his contributions to mechanisms of cell death. The scientific founders will join Mitokyne’s Scientific Advisory Board, which also includes founding member Jodi Nunnari, a Professor of Molecular & Cellular Biology at University of California, Davis.

“I am delighted that we were able to bring together a committed corporate partner in Astellas along with great investors and scientific advisors to help shape and build this company in such an exciting area,” said Kazumi Shiosaki, CEO and co-founder of Mitokyne. “Clearly Astellas and Mitokyne share a singular vision to become leaders in discovering and developing novel compounds that beneficially modulate mitochondrial function. Both companies will be able to contribute their complementary strengths to forge a robust partnership.”

The impact of this collaboration has been accounted in Astellas’ forecasts for fiscal year ending March 2014.

About AstellasAstellas Pharma Inc., located in Tokyo, Japan, is a pharmaceutical company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceuticals. Astellas has approximately 17,000 employees worldwide. The organization is committed to becoming a global category leader in Urology, Immunology (including Transplantation) and Infectious diseases, Oncology, Neuroscience and Diabetes Mellitus (DM) Complications and Kidney diseases. For more information on Astellas Pharma Inc., please visit the company website at www.astellas.com/en.

About MitokyneMitokyne is a biotech company focused on mitochondria-related drug discovery and development. The company is utilizing its innovative screening platform to identify and develop novel drugs that improve mitochondria function and provide treatments for genetic, metabolic and neurodegenerative disorders as well as diseases and conditions of aging. Mitokyne’s scientific founders and advisors include world-renowned leaders in biology and mitochondrial function.

About MPM CapitalMPM Capital is one of the world's largest life science-dedicated venture investors. With committed capital under management in excess of $2.6 billion, MPM Capital is uniquely structured to invest globally in healthcare innovation.

About Longwood Founders FundLongwood Founders Fund is a healthcare venture capital firm that founds, manages, and builds biotechnology companies. Longwood’s mission is to identify technologies and to found companies that will advance new therapeutics that can not only make a difference in the lives of patients worldwide, but also create significant value for investors. This is achieved by leveraging the management team’s history of successful biotechnology company formation and operational leadership.

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About MitochondriaMitochondria are present in almost every cell of the human body. The main function of mitochondria is generation of ATP as the energy for humans to function. In addition, mitochondria are involved in numerous other functions including fatty acid metabolism, reactive oxygen species (ROS) production/elimination, cell death regulation, and so on.

About Mitochondrial Diseases and Mitochondria-Related DiseasesMitochondrial diseases are caused by various mutations, acquired or inherited, in either mitochondrial DNA (mtDNA) or nuclear DNA (nDNA). Dysfunction of mitochondria can affect only certain tissues due to factors not yet fully understood. Affected patients suffer from muscular dysfunction, neurological dysfunction, cardiac dysfunction, ocular/otic dysfunction, metabolic dysfunction and so on. Typical onset in most cases is during early childhood, impairing a patient’s quality of life.

March 3, 2016

The Emerging Network of Mitochondria-Organelle Contacts.

April 8, 2014

Nature Biotechnology

Specialty firm Edison Pharmaceuticals of Mountain View, California, has entered a strategic alliance worth up to $4.3 billon with Dainippon Sumitomo Pharma (DSP) of Osaka, Japan, to develop drugs for inherited respiratory chain diseases of the mitochondria. Under terms of the deal, the companies will jointly expand Edison's pipeline, bringing…